Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

  • STATUS
    Recruiting
  • participants needed
    40
  • sponsor
    Vertex Pharmaceuticals Incorporated
Updated on 19 February 2024

Summary

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.

Details
Condition Alpha1-Antitrypsin Deficiency
Age 18years - 80years
Treatment Placebo, VX-864
Clinical Study IdentifierNCT04474197
SponsorVertex Pharmaceuticals Incorporated
Last Modified on19 February 2024

Eligibility

Yes No Not Sure

Inclusion Criteria

Subjects must have a PiZZ genotype confirmed at screening
Plasma AAT levels indicating severe deficiency at screening

Exclusion Criteria

History of a medical condition that could negatively impact the ability to complete the study
Solid organ, or hematological transplantation or is currently on a transplant list
History of use of gene therapy or RNAi therapy at any time previously
Other protocol defined Inclusion/Exclusion criteria may apply
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